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Asterias Biotherapeutics Announces Independent Data Monitoring Committee Completes Last Intermediate Review of AST-OPC1 Clinical Trial in Acute Spinal Cord Injury
- AST-OPC1 Safety Profile Continues To Be Favorable -
FREMONT, Calif., June 05, 2018 (GLOBE NEWSWIRE) -- Asterias Biotherapeutics, Inc. (NYSE American:AST), a biotechnology company dedicated to developing cell-based therapeutics to treat neurological conditions associated with demyelination and cellular immunotherapies to treat cancer, today announced that the independent Data Monitoring Committee (DMC) for the company’s SCiStar Phase 1/2a study of AST-OPC1 in acute spinal cord injury has completed a regularly scheduled review of the safety data from all subjects in the five study cohorts and has recommended that the SCiStar study continue according to protocol to its final data readout.
The DMC, comprised of an independent group of medical and scientific experts, has reviewed study data at regular intervals, with the primary responsibilities of reviewing and evaluating patient safety and efficacy data for the purpose of ensuring the safety of all patients enrolled in the study, the quality of the data collected, and the continued scientific validity of the study design.
“The DMC's recommendation to continue our SCiStar study without modification reaffirms the committee's previous safety reviews of AST-OPC1, and confirms the safety data to date from the high dose 10 and 20 million cell cohorts in the study,” said Dr. Edward Wirth, Chief Medical Officer of Asterias Biotherapeutics. “We sincerely thank the DMC for their diligent work in ensuring the quality and consistency of data from the SCiStar study as we approach the announcement of full results from the study.”
Asterias has dosed 25 subjects with AST-OPC1 in the SCiStar study and a total of 30 subjects including the five subjects from the previous Phase 1 safety trial. The results-to-date, which include subjects from the Phase 1 safety trial who have been followed for as long as seven years, continue to support the safety of AST-OPC1. In particular, there have been no serious, unexpected, adverse events related to AST-OPC1, the injection procedure, or the drug used for immunosuppression in any of the 30 subjects. Additionally, long-term follow up in the Phase 1 safety trial with annual magnetic resonance imaging scans through five years post-injection of AST-OPC1 has shown no evidence of adverse changes in any of the subjects treated with AST-OPC1.
In February 2018, Asterias reported additional results from the SCiStar study that continued to support AST-OPC1’s positive safety profile and its potential to durably engraft and help restore upper extremity motor function improvement to individuals with severe cervical spinal cord injuries.
Anticipated 2018 Data Readouts for the SCiStar Study
The company intends to report the following AST-OPC1 data readouts later this year:
- Six-month update for the entire SCiStar study, including Cohort 5 (AIS-B; 20M AST-OPC1 cells), late in the second quarter or early third quarter of 2018.
- 12-month update for Cohort 3 (AIS-A; 20M AST-OPC1 cells) and Cohort 4 (AIS-B; 10M AST-OPC1 cells) in the third quarter of 2018.
- 24-month update for Cohort 2 (AIS-A; 10M AST-OPC1 cells) in the third or fourth quarter of 2018.
- 12-month update for the entire SCiStar study, including Cohort 5, late in the fourth quarter of 2018 or early in the first quarter of 2019.
AST-OPC1, an oligodendrocyte progenitor cell population derived from human embryonic stem cells, has been shown in preclinical testing in animals and in vitro to have three potentially reparative functions that address the complex pathologies observed in demyelination disorders, such as spinal cord injuries, and multiple neurodegenerative diseases, including multiple sclerosis and white matter stroke. These potential reparative functions of AST-OPC1 include the production of neurotrophic factors, the stimulation of vascularization, and the induction of remyelination of denuded axons, all of which are critical for survival and regrowth of—and conduction of nerve impulses through—axons at the injury site.
Each year in the United States, more than 17,000 people suffer a severe, debilitating spinal cord injury. As of 2016, the National Spinal Cord Injury Statistical Center reported that approximately 4,500 of these new spinal cord injuries annually in the U. S. are AIS-A, AIS-B, or AIS-C patients with C-4 to C-7 spinal cord injuries (https://www.nscisc.uab.edu/). These injuries can be devastating to quality of life and ability to function independently. Lifetime healthcare costs for these patients can often approach $5 million. Improvements in arm, hand, and finger functional capabilities in these patients can result in meaningfully lower healthcare costs, significant improvements in quality of life, greater ability to engage in activities of daily living, and increased independence.
About the SCiStar Trial
The SCiStar trial completed enrollment in December 2017 and is an open-label, single-arm trial testing three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells in 25 subjects with subacute motor complete (AIS-A or AIS-B) cervical (C-4 to C-7) SCI. These individuals have essentially lost all movement below their injury site and experience severe paralysis of the upper and lower limbs. AIS-A subjects have lost all motor and sensory function below their injury site, while AIS-B subjects have lost all motor function but may have retained some minimal sensory function below their injury site. AST-OPC1 is administered 21 to 42 days post-injury. Subjects will be followed by neurological exams and imaging procedures to assess the safety and activity of the product.
Asterias has received a Strategic Partnerships Award grant from the California Institute for Regenerative Medicine, which provides $14.3 million of non-dilutive funding for the Phase 1/2a clinical trial and other product development activities for AST-OPC1.
Additional information on the Phase 1/2a trial, including trial sites, can be found at www.clinicaltrials.gov, using Identifier NCT02302157, and at the SCiStar Study Website (www.SCiStar-study.com).
About Asterias Biotherapeutics
Asterias Biotherapeutics, Inc. is a biotechnology company dedicated to developing cell-based therapeutics to treat neurological conditions associated with de-myelination and cellular immunotherapies to treat cancer. Asterias is presently focused on advancing three clinical-stage programs which have the potential to address areas of very high unmet medical need in the fields of neurology and oncology. AST-OPC1 (oligodendrocyte progenitor cells) is currently in a Phase 1/2a dose escalation clinical trial in spinal cord injury. AST-VAC2 (antigen-presenting allogeneic dendritic cells) represents a second generation, allogeneic cancer immunotherapy that also targets telomerase presenting tumor cells. The company's research partner, Cancer Research UK, plans to begin a first-in-human (FIH) clinical trial of AST-VAC2 in non-small cell lung cancer. AST-VAC1 (antigen-presenting autologous dendritic cells) is an autologous cancer immunotherapy targeting telomerase presenting tumor cells, with promising efficacy and safety data from a Phase 2 study in Acute Myeloid Leukemia (AML) which the company believes is proof of concept data for AST-VAC2. Additional information about Asterias can be found at www.asteriasbiotherapeutics.com.
Statements pertaining to future financial and/or operating and/or clinical research results, future growth in research, technology, clinical development, and potential opportunities for Asterias, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the businesses of Asterias, particularly those mentioned in the cautionary statements found in Asterias' filings with the Securities and Exchange Commission. Asterias disclaims any intent or obligation to update these forward-looking statements.